ProQR Therapeutics N.V. operates as a biopharmaceutical enterprise dedicated to discovering and developing RNA-based therapies designed to address genetic disorders. The company's pipeline includes two primary drug candidates in advanced clinical stages: Sepofarsen is currently in a Phase II/III clinical trial, known as the ILLUMINATE trial, targeting Leber Congenital Amaurosis 10 (LCA10). Ultevursen is also undergoing Phase II/III evaluation for the treatment of USH2A-mediated retinitis pigmentosa and Usher syndrome. Beyond its clinical programs, ProQR is actively developing its proprietary Axiomer RNA base-editing platform technology. Strategic collaborations are central to ProQR's strategy, evidenced by its licensing agreements with institutions such as Radboud University Medical Center, Inserm Transfert SA, Ionis Pharmaceuticals, Inc., and Leiden University Medical Center. Furthermore, a significant license and research partnership exists with Eli Lilly and Company, focused on the discovery, advancement, and commercialization of novel medicines for genetic conditions impacting the liver and nervous system. Established in 2012, ProQR Therapeutics N.V. maintains its corporate headquarters in Leiden, the Netherlands.
ProQR Announces Appointment of Chris Hart as Chief Data and AI Officer and Thomas Wolf to join the Board of Directors as Strategic AI Advisor
ProQR appointed Chris Hart, PhD, formerly VP of Data Science and AI/ML at Eli Lilly, as Chief Data and AI Officer, and Thomas Wolf, PhD, co-founder of Hugging Face, to its Board as strategic AI advisor.
The appointments support scaling AI across the Axiomer RNA editing platform, with AX-0811 — the first AI-discovered program in the clinic — on track for target engagement data in early January 2027.
The company expects up to 5 clinical data readouts over the next 12 months across 4 development programs.